CA2901328C - Compositions et methodes de traitement de la mps1 - Google Patents

Compositions et methodes de traitement de la mps1 Download PDF

Info

Publication number
CA2901328C
CA2901328C CA2901328A CA2901328A CA2901328C CA 2901328 C CA2901328 C CA 2901328C CA 2901328 A CA2901328 A CA 2901328A CA 2901328 A CA2901328 A CA 2901328A CA 2901328 C CA2901328 C CA 2901328C
Authority
CA
Canada
Prior art keywords
seq
raav
change
iduronidase
sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CA2901328A
Other languages
English (en)
Other versions
CA2901328A1 (fr
Inventor
James M. Wilson
Brittney L. Gurda
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania
Original Assignee
University of Pennsylvania
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Pennsylvania filed Critical University of Pennsylvania
Publication of CA2901328A1 publication Critical patent/CA2901328A1/fr
Application granted granted Critical
Publication of CA2901328C publication Critical patent/CA2901328C/fr
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

L'invention concerne un vecteur doté d'une cassette d'expression comprenant un gène hIDUA dont la séquence est SEQ ID NO : 1 ou une séquence identique à environ 95 % codant pour une alpha-L-iduronidase humaine fonctionnelle. Le vecteur peut être un vecteur de production ou un rAAV8. L'invention concerne aussi des compositions contenant ces vecteurs et des méthodes de traitement de la MPSI et des symptômes associés aux syndromes de Hurler, de Hurler-Scheie et de Scheie.
CA2901328A 2013-03-15 2014-03-13 Compositions et methodes de traitement de la mps1 Active CA2901328C (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361788724P 2013-03-15 2013-03-15
US61/788,724 2013-03-15
PCT/US2014/025509 WO2014151341A1 (fr) 2013-03-15 2014-03-13 Compositions et méthodes de traitement de la mpsi

Publications (2)

Publication Number Publication Date
CA2901328A1 CA2901328A1 (fr) 2014-09-25
CA2901328C true CA2901328C (fr) 2023-08-01

Family

ID=51580934

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2901328A Active CA2901328C (fr) 2013-03-15 2014-03-13 Compositions et methodes de traitement de la mps1

Country Status (21)

Country Link
US (3) US10137176B2 (fr)
EP (2) EP3747998B1 (fr)
JP (4) JP6591956B2 (fr)
KR (2) KR102346455B1 (fr)
CN (1) CN105026554B (fr)
AU (2) AU2014235096B2 (fr)
BR (1) BR112015021036A2 (fr)
CA (1) CA2901328C (fr)
CY (1) CY1123169T1 (fr)
DK (1) DK2984166T3 (fr)
ES (2) ES2805355T3 (fr)
HR (1) HRP20201063T1 (fr)
HU (1) HUE051373T2 (fr)
LT (1) LT2984166T (fr)
MX (2) MX386059B (fr)
PL (1) PL2984166T3 (fr)
PT (1) PT2984166T (fr)
RU (1) RU2708318C2 (fr)
SG (2) SG11201507507PA (fr)
SI (1) SI2984166T1 (fr)
WO (1) WO2014151341A1 (fr)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
US11555059B2 (en) * 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
EP4085934A1 (fr) * 2015-12-11 2022-11-09 The Trustees of The University of Pennsylvania Thérapie génique pour traiter l'hypercholestérolémie familiale
AU2016370590B2 (en) * 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
SG10201912935WA (en) 2015-12-15 2020-02-27 Genzyme Corp Adeno-associated viral vectors for treating mucolipidosis type ii
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
WO2017136533A1 (fr) * 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Méthodes pour traiter, diagnostiquer, et surveiller le traitement des mucopolysaccharidoses
CN108697774A (zh) * 2016-02-22 2018-10-23 北卡罗来纳大学教堂山分校 治疗mps i-相关失明的aav-idua载体
US11253612B2 (en) 2016-04-15 2022-02-22 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
IL262207B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
WO2018022511A1 (fr) * 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprenant un variant de la lécithine-cholestérol-acyl-transférase et leurs utilisations
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
CA3034089A1 (fr) 2016-08-18 2018-02-22 The Regents Of The University Of California Ingenierie genomique de crispr-cas par l'intermediaire d'un systeme d'administration d'aav modulaire
EP3562494A4 (fr) 2016-12-30 2020-08-19 The Trustees Of The University Of Pennsylvania Thérapie génique pour le traitement de la phénylcétonurie
CA3068328A1 (fr) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Therapie genique mediee par aav9 pour traiter la mucopolysaccharidose de type i
EP3684938A1 (fr) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Thérapie génique pour le traitement de la mucopolysaccharidose de type ii
CN107699590A (zh) * 2017-10-13 2018-02-16 成都中医药大学 一种制备重组人α‑L‑艾杜糖醛酸酶的方法
EA202091352A1 (ru) 2017-11-30 2020-08-27 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Генотерапия мукополисахаридоза iiib
WO2019152841A1 (fr) 2018-02-01 2019-08-08 Homology Medicines, Inc. Compositions de virus adéno-associés pour transfert de gène de pah et procédés d'utilisation
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
CA3099958A1 (fr) 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions et methodes de traitement de la dystrophie musculaire de duchenne
KR20210008501A (ko) 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
CA3106078A1 (fr) 2018-07-17 2020-01-23 Junghun Lee Traitement de neuropathie avec des constructions d'adn exprimant des isoformes d'igf-1
CN119390787A (zh) 2019-01-31 2025-02-07 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
CA3183153A1 (fr) 2020-06-17 2021-12-23 Christian HINDERER Compositions et methodes pour le traitement de patients de therapie genique
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
KR102590276B1 (ko) * 2021-01-04 2023-10-16 부산대학교 산학협력단 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
ES3061188T3 (en) 2021-09-08 2026-03-31 Covestro Deutschland Ag Succinic acid based polyester polyols
CN114181318B (zh) * 2021-11-08 2023-08-01 四川大学 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
WO2023087019A2 (fr) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions pour la réduction spécifique de drg de l'expression de transgènes
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CA3247109A1 (fr) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions et méthodes de traitement d'un cancer du sein métastatique récepteur 2 de facteur de croissance épidermique humain positif et d'autres cancers
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
JP2026522354A (ja) 2023-06-12 2026-07-07 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉｉｂのためのａａｖ遺伝子療法
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025129157A1 (fr) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Thérapie génique pour le traitement de la maladie de canavan

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6149909A (en) * 1995-06-23 2000-11-21 Women's And Children's Hospital Synthetic α-L-iduronidase and genetic sequences encoding same
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
CA2264482A1 (fr) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania Procede inductible de production de virus adeno-associes recombines au moyen de la polymerase t7
WO1999058691A2 (fr) 1998-05-13 1999-11-18 Harbor-Ucla (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
RU2196988C2 (ru) 2000-04-19 2003-01-20 Государственный новосибирский областной клинический диагностический центр Способ диагностики мукополисахаридозов
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
US7155804B2 (en) 2003-09-17 2007-01-02 Moog Inc. Method of forming an electric motor
EP1668143B1 (fr) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Variantes des virus associes aux adenovirus (aav), sequences, vecteurs les contenant, et leur utilisation
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20090062144A1 (en) 2007-04-03 2009-03-05 Nancy Lan Guo Gene signature for prognosis and diagnosis of lung cancer
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
JP2014503173A (ja) * 2009-11-05 2014-02-13 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 制御された発現系
EP2333074A1 (fr) * 2009-12-14 2011-06-15 Robert Steinfeld Substances et procédés pour le traitement de maladies liées au stockage lysosomal
WO2011126808A2 (fr) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Système d'ablation de transgène induit pharmacologiquement
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (fr) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vecteurs et séquences pour le traitement de maladies
CN103180445B (zh) * 2010-10-22 2018-02-16 库尔纳公司 通过抑制α‑L‑艾杜糖醛酸酶(IDUA)的天然反义转录物而治疗IDUA相关疾病
CN103502458B (zh) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
HUE051612T2 (hu) 2012-07-11 2021-03-01 Sangamo Therapeutics Inc Eljárások és készítmények lizoszomális tárolási betegségek kezelésére
CA2892027A1 (fr) 2012-11-21 2014-05-30 Rqx Pharmaceuticals, Inc. Antibiotiques macrocycliques a large spectre
WO2014125647A1 (fr) 2013-02-18 2014-08-21 富士通オプティカルコンポーネンツ株式会社 Dispositif photorécepteur
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
JP2016523835A (ja) 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
WO2016049230A1 (fr) 2014-09-24 2016-03-31 City Of Hope Variants de vecteur de virus adénoassocié pour une édition de haute efficacité du génome et procédés correspondants
CN107002096A (zh) 2014-10-06 2017-08-01 阿罗根有限公司 基于aav的基因疗法
KR20220082100A (ko) 2015-05-15 2022-06-16 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 치료적 전달
CN107922932A (zh) 2015-08-06 2018-04-17 中央研究院 用于酶置换疗法的改造酶
AU2016341428B2 (en) 2015-10-23 2021-12-02 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
EP3992283A1 (fr) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif d'aavrh10
EP3387138B2 (fr) 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Méthode de purification évolutive pour virus adéno-associé 9 (aav9)
WO2017100674A1 (fr) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif pour virus adéno-associé (aav) 1
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
EP3402533B1 (fr) 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Méthodes et compositions pour le traitement d'une maladie neurologique
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
CA3068328A1 (fr) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Therapie genique mediee par aav9 pour traiter la mucopolysaccharidose de type i

Also Published As

Publication number Publication date
ES2991471T3 (es) 2024-12-03
RU2015144234A (ru) 2017-04-24
MX2015012739A (es) 2016-02-19
CY1123169T1 (el) 2021-10-29
JP6591956B2 (ja) 2019-10-16
AU2014235096B2 (en) 2020-03-19
RU2708318C2 (ru) 2019-12-05
EP2984166B1 (fr) 2020-04-22
PT2984166T (pt) 2020-07-16
HUE051373T2 (hu) 2021-03-01
SI2984166T1 (sl) 2020-09-30
KR102346455B1 (ko) 2022-01-04
WO2014151341A1 (fr) 2014-09-25
US10792343B2 (en) 2020-10-06
US20200397872A1 (en) 2020-12-24
KR20220005599A (ko) 2022-01-13
LT2984166T (lt) 2020-08-10
CN105026554A (zh) 2015-11-04
US20160000887A1 (en) 2016-01-07
EP2984166A1 (fr) 2016-02-17
EP3747998A1 (fr) 2020-12-09
CN105026554B (zh) 2020-08-11
SG11201507507PA (en) 2015-10-29
KR20150129678A (ko) 2015-11-20
PL2984166T3 (pl) 2021-01-11
CA2901328A1 (fr) 2014-09-25
EP3747998A9 (fr) 2021-08-04
MX2021010918A (es) 2021-10-01
JP7553527B2 (ja) 2024-09-18
SG10201707319UA (en) 2017-10-30
AU2014235096A1 (en) 2015-09-10
JP7168621B2 (ja) 2022-11-09
HK1221735A1 (en) 2017-06-09
HRP20201063T1 (hr) 2020-11-27
JP2016512683A (ja) 2016-05-09
BR112015021036A2 (pt) 2017-10-10
KR102581652B1 (ko) 2023-09-22
MX386059B (es) 2025-03-18
EP3747998B1 (fr) 2024-07-24
US20190111116A1 (en) 2019-04-18
AU2020203826B2 (en) 2022-09-15
US10137176B2 (en) 2018-11-27
EP2984166A4 (fr) 2016-09-07
JP2020202863A (ja) 2020-12-24
JP2023011752A (ja) 2023-01-24
JP2019134717A (ja) 2019-08-15
AU2020203826A1 (en) 2020-07-02
ES2805355T3 (es) 2021-02-11
DK2984166T3 (da) 2020-07-20

Similar Documents

Publication Publication Date Title
JP7553527B2 (ja) Ｍｐｓ１を治療するための組成物および方法
JP2022513034A (ja) 神経セロイドリポフスチン症の遺伝子治療
JP2022523766A (ja) Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
TW202229560A (zh) 治療法布瑞氏症之組成物及方法
JP2022512589A (ja) Ｇｍ１ガングリオシドーシスの治療に有用な組成物
TW202045728A (zh) 用於治療克拉培氏病之組成物
JP2024511024A (ja) ２１－ヒドロキシラーゼ欠損症の遺伝子治療
US20230190966A1 (en) Compositions useful for treating gm1 gangliosidosis
HK40042501A (en) Compositions for treating mpsi
HK40042501B (en) Compositions for treating mpsi
HK1221735B (en) Compositions for treating mpsi
US20230136699A1 (en) Gene therapy for maple syrup urine disease

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20190313

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 11TH ANNIV.) - STANDARD

Year of fee payment: 11

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250220

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20250220

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250220

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 12TH ANNIV.) - STANDARD

Year of fee payment: 12

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260217

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260217